The autosomal dominant optic atrophy treatment market is experiencing significant expansion, driven by increasing disease awareness, rising prevalence, and R&D in precision medicine from key ...

By clicking “Accept All Cookies”, you agree to the storing of cookies on your device to enhance site navigation, analyze site usage, and assist in our marketing efforts. You accept for the main domain ...

Such a cobblestone tongue is a telltale sign of a rare genetic condition called Cowden syndrome. It's caused by inherited ...

Ultimately, Stoke Therapeutics is that classic “intriguing biotech” --hurdling to one of those proverbial “moments of truth” ...

PKD is a genetic disorder marked by kidney cysts and progressive damage. Discover the science, treatment guidelines, and research driving future care.

A large longitudinal study found that pesticide exposure and caffeinated soda intake were linked to more severe motor ...

About the Phase 2 IGNITE Clinical Trial and ILLUMINATE Natural History Study ...

In a nutshell Researchers analyzed over 4,000 proteins in the cerebrospinal fluid of people with genetic forms of frontotemporal dementia (FTD), identifying distinct protein networks linked to disease ...

Most forms of NDM and MODY are caused by autosomal dominant mutations, meaning they can be passed on to children when only one parent carries the gene for the disease. In these cases, the parent ...

Gene therapies challenge ERT dominance in Gaucher’s disease as new data reveals strategic insights, key players, and future ...